Links to some of the very latest research taking place for lysosomal diseases.
- FDA approves Vimizim to treat Morquio A
- BioMarin Vimizim accepted by FDA for review
- BioMarin begins ERT Trials for Batten Disease
- LDNZ grants $5,000 to UK MPS Society to support Genistein clinical trail for Sanflippo III
- BioMarin submits Vimizim BLA to U.S. FDA for the treatment of MPS IVA
- Lysogene receives US Orphan Designation for its lean intracerebral Gene Therapy for Sanflippo A